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Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

A new genome editing strategy can normalize the β-globin:α-globin balance in human hematopoietic stem cells from patients with β-thalassemia and restore functional adult hemoglobin tetramers in patient-derived red blood cells.

M. Kyle Cromer
Joab Camarena
Matthew H. Porteus
Research18 Mar 2021
Nature Medicine

Volume: 27, P: 677-687

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Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

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